Rhythm Pharmaceuticals

What Rhythm Pharmaceuticals does

Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company headquartered in Boston, Massachusetts (NASDAQ: RYTM) that develops and commercializes peptide therapeutics targeting the melanocortin-4 receptor (MC4R) pathway for rare neuroendocrine diseases characterized by hyperphagia and severe obesity. The company's lead product, IMCIVREE (setmelanotide), is a first-in-class MC4R agonist currently approved by the FDA for chronic weight management in patients with obesity due to POMC, PCSK1, or LEPR deficiency and Bardet-Biedl syndrome, and received FDA approval in March 2026 for acquired hypothalamic obesity — making it the first and only FDA-approved therapy for that indication. The pipeline extends to an oral MC4R-specific agonist (bivamelagon/LB54640, Phase 2 SIGNAL trial) and a weekly MC4R agonist (RM-718, Phase 2 in Prader-Willi syndrome), with a stated goal of moving beyond the first-generation daily subcutaneous injection.

Name

Rhythm Pharmaceuticals

Legal name

Rhythm Pharmaceuticals, Inc.

Website

https://rhythmtx.com

Company type

Public

Founded year

2008

Operating status

Operating

Headcount range

251–500 employees

Ownership category

akta.pro rank

Product category

Rare Disease Therapeutics

NAICS

Pharmaceutical Preparation Manufacturing (325412)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Neurology & CNS Specialty Pharmaceuticals (HLAIACAC)

akta.pro secondary industry

Cardiometabolic Pharmaceuticals (Cardiovascular/Diabetes/Obesity) (HLAIAAAE)

Keywords

• Rare disease therapeutics
• Obesity treatment drugs
• Peptide therapeutics
• Biopharmaceutical products
• MC4R receptor agonists

Headquarters

HQ city

Boston

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2C

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Marketing or Sales, Operations

Revenue model

1. IMCIVREE (setmelanotide) Product Sales: Global net product sales of IMCIVREE for rare genetic obesity conditions. Revenue generated from direct product sales through specialty pharmacy distribution channels. Q1 2026 revenue of $60.1 million with 61% from U.S. and 39% international. Full-year 2025 revenue of approximately $194 million, representing 50% year-over-year growth.

Pricing tiers

Go-to-market motion1 record

Distribution channels2 records

Marketing channels7 records

Core offering

Rhythm Pharmaceuticals develops and commercializes peptide therapeutics targeting the melanocortin-4 receptor (MC4R) pathway for the treatment of rare neuroendocrine diseases. Its lead product, IMCIVREE (setmelanotide), is an FDA-approved injection for chronic weight management in patients with monogenic or syndromic obesity (POMC, PCSK1, LEPR deficiency, Bardet-Biedl syndrome) and acquired hypothalamic obesity.

Differentiator

Problem solved

Functional benefit

Brands

• IMCIVREE: FDA-approved therapy (setmelanotide) for chronic weight management in patients with rare genetic obesity conditions including POMC, PCSK1, LEPR deficiency and Bardet-Biedl syndrome
• Rhythm InTune
• GOLD Academy
• LEAD for Rare Obesity
• Uncovering Rare Obesity
• Rhythm Imtune

Products and services

• IMCIVREE (setmelanotide) FDA-approved MC4R agonist injection for chronic weight management in patients 6 years and older with monogenic or syndromic obesity due to POMC, PCSK1, or LEPR deficiency and Bardet-Biedl syndrome. Now also approved for acquired hypothalamic obesity in adults and pediatric patients aged 4 and older. Distributed through specialty pharmacy channels.

Quantifiable outcome

• 18.4% placebo-adjusted BMI reduction at 52 weeks in acquired hypothalamic obesity (TRANSCEND trial)
• +4 more outcomes

Named customers4 records

Segments3 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Three partnerships are on record, tiered strategic and core.

• LG Chem Life SciencesstrategicStrategic or Co-development Partner · 1 January 2024Acquired oral MC4R agonist LB54640 (now named bivamelagon) from LG Chem Life Sciences. This next-generation oral therapy is being developed as a potential treatment for acquired hypothalamic obesity and other rare MC4R pathway diseases.
• Xinvento BVstrategicStrategic or Co-development Partner · 27 February 2023Rhythm Pharmaceuticals BV, a Dutch subsidiary of US-based Rhythm Pharmaceuticals, acquired biotech company Xinvento BV in Amsterdam for up to $211 million. The acquisition focuses on developing therapies for congenital hyperinsulinism (CHI), with key contributions from Xinvento's founder Claudine van der Sande. The deal includes upfront, milestone, and sales-based payments.
• PANTHERx Rare PharmacycoreChannel Partner/ Reseller/ Distributor · 20 March 2020PANTHERx Rare Pharmacy is the exclusive U.S. specialty pharmacy distribution partner for IMCIVREE. The partnership began in 2020 for rare obesity indications (Bardet-Biedl syndrome and POMC, PCSK1, LEPR deficiency) and was expanded in March 2026 to include the new acquired hypothalamic obesity indication. PANTHERx handles specialty distribution, patient support, and reimbursement services for the drug.

Scale indicators10 records

Recent moves7 records

Expansion highlights6 records

Broad incumbents

• Vertex Pharmaceuticals: Larger rare-disease innovator with franchise in CF, sickle cell and pain. Comparable to Rhythm as a science-driven, single-mechanism-focused rare disease company with premium orphan economics and expanding label strategy.
• Sanofi (Genzyme): Large-cap pharma with rare disease franchise (Genzyme legacy) including rare metabolic and endocrine disorder therapies. Comparable to Rhythm as a rare-disease benchmark; CEO David Meeker and several other Rhythm executives are Genzyme alumni, and Sanofi Genzyme is the prior employer of multiple key Rhythm leaders.
• Novo Nordisk: Global leader in obesity therapeutics (Wegovy/Saxenda) and rare endocrine disease player. Relevant broad incumbent because of category overlap (obesity therapeutics), large commercial muscle, and potential interest in expanding GLP-1 franchises into rare genetic obesity indications.

Emerging players

• Crinetics Pharmaceuticals: Clinical-stage rare endocrine disease developer (acromegaly, Cushing's, congenital hyperinsulinism programs). Direct overlap with Rhythm's MC4R pathway and rare endocrine disease focus, and direct competitor in the congenital hyperinsulinism indication Rhythm entered via Xinvento.
• Soleno Therapeutics: Developer of DCCR for Prader-Willi syndrome hyperphagia. Direct emerging competitor to setmelanotide in PWS — a key Phase 3 indication Rhythm is advancing — making it the most direct therapeutic-overlap peer.
• Aardvark Therapeutics: Clinical-stage developer of ARD-101, an oral MC4R agonist for rare and common obesity indications. Direct emerging competitor to Rhythm in the MC4R pathway space and to the oral bivamelagon program in particular.

Direct peers

• Alnylam Pharmaceuticals: Specialty biopharma commercializing RNA-based therapies for rare genetic diseases with similar orphan-drug commercial economics, specialty distribution, and pipeline expansion via platform technology.
• Ionis Pharmaceuticals: Specialty biopharma with antisense oligonucleotide platform targeting rare neurological and metabolic diseases. Comparable to Rhythm in rare disease pipeline depth, specialty commercial model, and multi-indication strategy from a single mechanism platform.
• Ultragenyx Pharmaceutical: Late-stage rare disease biotech commercializing treatments for ultra-rare metabolic and endocrine disorders (Crysvita, Dojolvi). Comparable to Rhythm in patient identification challenges, specialty distribution, and pipeline of orphan endocrine/metabolic assets.
• BioMarin Pharmaceutical: Commercial-stage rare disease biopharma with a multi-indication orphan drug franchise (e.g., Vimizim, Naglazyme, Voxzogo). Directly comparable to Rhythm in business model: focused rare disease pipeline, specialty/rare distribution, premium orphan pricing, and similar reliance on individual product success.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks7 records

Key highlights7 records

Customer concentration

• Xx.com/rhythmpharma
• LinkedInlinkedin.com/company/rhythm-pharmaceuticals-inc-

Revenue estimate

Valuation estimate

Number of profiles

Profiles12 records

Subsidiaries1 record

Funding overview

Funding rounds12 records

Investors8 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A1 record

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Rhythm Pharmaceuticals